PTC124
PTC124 allows read-through of premature stop codons in mRNA, enabling proper protein translation. This compound is used to treat Duchenne muscular dystrophy (DMD) and cystic fibrosis as well as other nonsense mutation disorders.
References PubMed ID：:http://www.ncbi.nlm.nih.gov/pubmed/18955472
| Cas No. |
775304-57-9 |
|---|---|
| Purity |
≥98% |
| Formula |
C15H9FN2O3 |
| Formula Wt. |
284.25 |
| IUPAC Name |
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| Synonym |
PTC-124, Ataluren, Translarna™ |
Bushby K, Finkel R, Wong B, et al. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve. 2014 Oct;50(4):477-87. PMID: 25042182.
Ryan NJ. Ataluren: first global approval. Drugs. 2014 Sep;74(14):1709-14. PMID: 25193627.
Nagel-Wolfrum K, Möller F, Penner I, et al. Translational read-through as an alternative approach for ocular gene therapy of retinal dystrophies caused by in-frame nonsense mutations. Vis Neurosci. 2014 Sep;31(4-5):309-16. PMID: 24912600.
